PERSEPT 3: A phase 3 clinical trial to evaluate the haemostatic efficacy of eptacog beta (recombinant human FVIIa) in perioperative care in subjects with haemophilia A or B with inhibitors.

Escobar, Miguel;Luck, James;Averianov, Yevhenii;Ducore, Jonathan;Hermans, Cédric;et.al.
(2021) Haemophilia (Print) — Vol. 27, n° 6, p. 911-920 (2021)

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  • Escobar, Miguelorcid-logo
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  • Luck, James
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  • Averianov, Yevhenii
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  • Ducore, Jonathan
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  • Hermans, CédricUCLouvain
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Abstract
INTRODUCTION: Surgical procedures in persons with haemophilia A or B with inhibitors (PwHABI) require the use of bypassing agents (BPA) and carry a high risk of complications. Historically, only two BPAs have been available; these are reported to have variable responses. AIM: To prospectively evaluate the efficacy and safety of a new bypassing agent, human recombinant factor VIIa (eptacog beta) in elective surgical procedures in PwHABI in a phase 3 clinical trial, PERSEPT 3. METHODS: Subjects were administered 200 µg/kg (major procedures) or 75 µg/kg eptacog beta (minor procedures) immediately prior to the initial surgical incision; subsequent 75 µg/kg doses were administered to achieve postoperative haemostasis and wound healing. Efficacy was assessed on a 4-point haemostatic scale during the intra- and postoperative periods. Anti-drug antibodies, thrombotic events and changes in clinical/laboratory parameters were monitored throughout the perioperative period. RESULTS: Twelve subjects underwent six major and six minor procedures. The primary efficacy endpoint success proportion was 100% (95% CI: 47.8%-100%) for minor procedures and 66.7% (95% CI: 22.3%-95.7%) for major procedures; 81.8% (95% CI: 48.2%-97.7%) of the procedures were considered successful using eptacog beta. There was one death due to bleeding from a nonsurgical site; this was assessed as unlikely related to eptacog beta. No thrombotic events or anti-eptacog beta antibodies were reported. CONCLUSION: Two eptacog beta dosing regimens in PwHABI undergoing major and minor surgical procedures were well-tolerated, and the majority of procedures were successful based on surgeon/investigator assessments. Eptacog beta offers clinicians a new potential therapeutic option for procedures in PwHABI.
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Escobar, M., Luck, J., Averianov, Y., Ducore, J., Fernández, M. F. L., Giermasz, A., Hart, D. P., Journeycake, J., Kessler, C., Leissinger, C., Mahlangu, J., Martinez, L. V., Miesbach, W., Mitha, I. H., Quon, D., Reding, M. T., Schved, J.-F., Stasyshyn, O., Vilchevska, K. V., et al. (2021). PERSEPT 3: A phase 3 clinical trial to evaluate the haemostatic efficacy of eptacog beta (recombinant human FVIIa) in perioperative care in subjects with haemophilia A or B with inhibitors. Haemophilia (Print), 27(6), 911-920. https://doi.org/10.1111/hae.14418 (Original work published 2021)